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Hematology

Promising role of voxelotor in managing sickle cell disease in children: a narrative review

Amit Agrawal, Gaurav Jadon, Japna Singh, Dalwinder Janjua

Clin Exp Pediatr. 2025;68(2):106-114. Published online November 13, 2024

Voxelotor has promising ability to increase hemoglobin levels and reduce hemolysis markers in patients with sickle cell disease (SCD). Several preclinical and phase II/III trials have demonstrated its efficacy, dose-dependent responses, and tolerability in children. Ongoing trials are assessing its safety and effectiveness in various populations, including children younger than 12 years. These findings suggest its potential as a disease-modifying drug, warranting further exploration of its role in SCD management.

DOI: https://doi.org/10.3345/cep.2024.00500

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